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About the Regulatory Profession

The regulatory function is vital in making safe and effective healthcare products available worldwide. Individuals who ensure regulatory compliance and prepare submissions, as well as those whose main job function is clinical affairs or quality assurance are all considered regulatory professionals.

Regulatory Code of Ethics

One of our most valuable contributions to the profession is the Regulatory Code of Ethics. The Code of Ethics provides regulatory professionals with core values that hold them to the highest standards of professional conduct.

Regulatory Competency Framework

Like all professions, regulatory is based on a shared set of competencies. The Regulatory Competency Framework describes the essential elements of what is required of regulatory professionals at four major career and professional levels.

Regulatory Convergence

Join the brightest minds in regulatory at the annual Regulatory Convergence. See the global regulatory community in action. Intensive workshops. Topical sessions. Meet ups with regulators. This is where it all comes together.

FDA Approves First Treatment for Chronic Graft Versus Host Disease

Posted 02 August 2017 | By Zachary Brennan

The US Food and Drug Administration (FDA) on Wednesday expanded the approval of AbbVie’s Imbruvica (ibrutinib) for the treatment of adult patients with chronic graft versus host disease (cGVHD) after the failure of one or more treatments. This is the first FDA-approved therapy for the treatment of cGVHD.

Background

cGVHD is a life-threatening condition that can occur in patients undergoing hematopoietic stem cell transplantation (HSCT) for certain blood or bone marrow cancers.

According to FDA, cGVHD occurs when cells from the stem cell transplant attack healthy cells in a patient’s tissues, and the condition is estimated to occur in 30-70% of all patients who receive HSCT.

AbbVie acquired Imbruvica, a kinase inhibitor, when it purchased Pharmacyclics for $21 billion in 2015. The treatment was previously approved for certain indications in treating chronic lymphocytic leukemia, Waldenström’s macroglobulinemia and marginal zone lymphoma, as well as mantle cell lymphoma.

Approval

The efficacy and safety of Imbruvica for the treatment of cGVHD were studied in a single-arm trial of 42 patients with cGVHD whose symptoms persisted despite standard treatment with corticosteroids.

In the trial, 67% of patients experienced improvements in their cGVHD symptoms, and in 48% of patients in the trial, the improvement of symptoms lasted for up to five months or longer. Common adverse events included fatigue, bruising, diarrhea, thrombocytopenia, stomatitis, muscle spasms, nausea, hemorrhage, anemia and pneumonia. Atrial fibrillation occurred in one patient and 24% of patients receiving Imbruvica in the cGVHD trial discontinued treatment due to adverse reactions.

Richard Pazdur, director of FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products, said: "Patients with cGVHD who do not respond to other forms of therapy—typically corticosteroids to suppress their immune system—now have a treatment option specifically indicated to treat their condition. This approval highlights how a known treatment for cancer is finding a new use in treating a serious and life-threatening condition that may occur in patients with blood cancer who receive a stem cell transplant."

Lori Styles, senior medical director at Abbvie’s Pharmacyclics added: "The FDA's approval of IMBRUVICA in chronic graft-versus-host-disease after failure of one or more lines of systemic therapy addresses an area of high unmet medical need for patients and marks the first approved use for the therapy outside of blood cancers."